An analysis of 2,684 matched patients in U.S. electronic health records linked acoramidis to a 27% lower risk of cardiovascular hospitalization than tafamidis. It is retrospective, follow-up is short, and it is BridgeBio's second such release in three weeks.
BridgeBio Pharma used a Sunday afternoon release to take its fight for heart-failure patients to a new arena: medical records rather than clinical trials.
The company said an independent study of U.S. electronic health records found that patients with transthyretin amyloid cardiomyopathy, known as ATTR-CM, who took its drug acoramidis had significantly better clinical outcomes than those who took tafamidis, the established treatment sold by Pfizer. The analysis drew on the Epic Cosmos database and compared 2,684 patients matched on their characteristics. Acoramidis was associated with a 27% lower risk of hospitalization for cardiovascular causes.
What kind of evidence this is
The study is retrospective. It looks back at records of patients whose doctors chose a treatment, rather than randomly assigning them to one. Statistical matching on observed characteristics reduces, but cannot eliminate, the chance that the two groups differed in ways that also affected their outcomes.
BridgeBio said so itself, noting that the study has limitations, including the possibility of residual confounding and relatively short follow-up.
Real-world comparisons still carry commercial weight. Cardiologists and insurers choosing between two approved therapies look at more than the original trials, and a records-based study that favors one drug becomes part of that conversation.
A repeated message
This is the second real-world comparison BridgeBio has promoted in less than three weeks; it issued a similar release on Sept. 23. A steady drumbeat of such data is a familiar strategy for a challenger trying to take share from an incumbent, and repetition does not add evidentiary weight on its own.
BridgeBio shares closed at $65.35 on Friday, giving the company a market value of about $12.8 billion.
Two readings
One reading is that the accumulation of real-world evidence is starting to matter. Two analyses in three weeks point in the same direction, the effect size on hospitalization is meaningful, and prescribers are making choices without a head-to-head trial.
The other is that the evidence has not changed. A retrospective, company-promoted study with short follow-up cannot establish that one drug is better than another, and Pfizer's product remains the established treatment.
What to watch
Publication of the analysis in a peer-reviewed journal, any response from Pfizer, and prescription-share data in BridgeBio's next quarterly report will show whether the message is changing behavior.